{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,19]],"date-time":"2026-09-19T21:27:14Z","timestamp":1789853234859,"version":"4.0.1"},"reference-count":131,"publisher":"Public Library of Science (PLoS)","issue":"2","license":[{"start":{"date-parts":[[2019,2,22]],"date-time":"2019-02-22T00:00:00Z","timestamp":1550793600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["www.plosone.org"],"crossmark-restriction":false},"short-container-title":["PLoS ONE"],"DOI":"10.1371\/journal.pone.0212198","type":"journal-article","created":{"date-parts":[[2019,2,22]],"date-time":"2019-02-22T18:45:43Z","timestamp":1550861143000},"page":"e0212198","update-policy":"https:\/\/doi.org\/10.1371\/journal.pone.corrections_policy","source":"Crossref","is-referenced-by-count":39,"title":["CRISPR-cas gene-editing as plausible treatment of neuromuscular and nucleotide-repeat-expansion diseases: A systematic review"],"prefix":"10.1371","volume":"14","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-0813-0005","authenticated-orcid":true,"given":"Haris","family":"Baba\u010di\u0107","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aditi","family":"Mehta","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4151-3916","authenticated-orcid":true,"given":"Olivia","family":"Merkel","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Benedikt","family":"Schoser","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"340","published-online":{"date-parts":[[2019,2,22]]},"reference":[{"key":"ref1","doi-asserted-by":"crossref","first-page":"227","DOI":"10.1146\/annurev-biochem-060815-014607","article-title":"CRISPR\/Cas9 in Genome Editing and Beyond","volume":"85","author":"H Wang","year":"2016","journal-title":"Annual review of biochemistry"},{"issue":"9","key":"ref2","doi-asserted-by":"crossref","first-page":"a016428","DOI":"10.1101\/cshperspect.a016428","article-title":"Sources of DNA double-strand breaks and models of recombinational DNA repair","volume":"6","author":"A Mehta","year":"2014","journal-title":"Cold Spring Harbor perspectives in biology"},{"issue":"3","key":"ref3","doi-asserted-by":"crossref","first-page":"519","DOI":"10.1093\/genetics\/122.3.519","article-title":"Genetic and physical analysis of double-strand break repair and recombination in Saccharomyces cerevisiae","volume":"122","author":"N Rudin","year":"1989","journal-title":"Genetics"},{"issue":"4","key":"ref4","doi-asserted-by":"crossref","first-page":"1968","DOI":"10.1128\/MCB.15.4.1968","article-title":"Induction of homologous recombination in mammalian chromosomes by using the I-SceI system of Saccharomyces cerevisiae","volume":"15","author":"A Choulika","year":"1995","journal-title":"Molecular and cellular biology"},{"issue":"12","key":"ref5","doi-asserted-by":"crossref","first-page":"8096","DOI":"10.1128\/MCB.14.12.8096","article-title":"Introduction of double-strand breaks into the genome of mouse cells by expression of a rare-cutting endonuclease","volume":"14","author":"P Rouet","year":"1994","journal-title":"Molecular and cellular biology"},{"issue":"13","key":"ref6","doi-asserted-by":"crossref","first-page":"6064","DOI":"10.1073\/pnas.91.13.6064","article-title":"Expression of a site-specific endonuclease stimulates homologous recombination in mammalian cells","volume":"91","author":"P Rouet","year":"1994","journal-title":"Proceedings of the National Academy of Sciences of the United States of America"},{"issue":"4","key":"ref7","doi-asserted-by":"crossref","first-page":"347","DOI":"10.1038\/nbt.2842","article-title":"CRISPR-Cas systems for editing, regulating and targeting genomes","volume":"32","author":"JD Sander","year":"2014","journal-title":"Nature biotechnology"},{"key":"ref8","doi-asserted-by":"crossref","first-page":"120","DOI":"10.1016\/j.jconrel.2014.12.036","article-title":"Current and future delivery systems for engineered nucleases: ZFN, TALEN and RGEN","volume":"205","author":"Q Ul Ain","year":"2015","journal-title":"Journal of controlled release: official journal of the Controlled Release Society"},{"issue":"1","key":"ref9","doi-asserted-by":"crossref","first-page":"15","DOI":"10.1042\/BJ20140295","article-title":"TALEN-mediated genome editing: prospects and perspectives","volume":"462","author":"DA Wright","year":"2014","journal-title":"The Biochemical journal"},{"key":"ref10","article-title":"Edited course of biomedical research: leaping forward with CRISPR","author":"PJ Collins","year":"2017","journal-title":"Pharmacological research"},{"issue":"2","key":"ref11","doi-asserted-by":"crossref","first-page":"174","DOI":"10.1007\/s00239-004-0046-3","article-title":"Intervening sequences of regularly spaced prokaryotic repeats derive from foreign genetic elements","volume":"60","author":"FJ Mojica","year":"2005","journal-title":"Journal of molecular evolution"},{"issue":"Pt 3","key":"ref12","doi-asserted-by":"crossref","first-page":"653","DOI":"10.1099\/mic.0.27437-0","article-title":"CRISPR elements in Yersinia pestis acquire new repeats by preferential uptake of bacteriophage DNA, and provide additional tools for evolutionary studies","volume":"151","author":"C Pourcel","year":"2005","journal-title":"Microbiology (Reading, England)"},{"issue":"5819","key":"ref13","doi-asserted-by":"crossref","first-page":"1709","DOI":"10.1126\/science.1138140","article-title":"CRISPR provides acquired resistance against viruses in prokaryotes","volume":"315","author":"R Barrangou","year":"2007","journal-title":"Science (New York, NY)"},{"issue":"7320","key":"ref14","doi-asserted-by":"crossref","first-page":"67","DOI":"10.1038\/nature09523","article-title":"The CRISPR\/Cas bacterial immune system cleaves bacteriophage and plasmid DNA","volume":"468","author":"JE Garneau","year":"2010","journal-title":"Nature"},{"issue":"5","key":"ref15","doi-asserted-by":"crossref","first-page":"945","DOI":"10.1016\/j.cell.2009.07.040","article-title":"RNA-guided RNA cleavage by a CRISPR RNA-Cas protein complex","volume":"139","author":"CR Hale","year":"2009","journal-title":"Cell"},{"issue":"2","key":"ref16","doi-asserted-by":"crossref","first-page":"156","DOI":"10.4161\/rna.27990","article-title":"CRISPR adaptive immune systems of Archaea","volume":"11","author":"G Vestergaard","year":"2014","journal-title":"RNA biology"},{"issue":"39","key":"ref17","doi-asserted-by":"crossref","first-page":"E2579","DOI":"10.1073\/pnas.1208507109","article-title":"Cas9-crRNA ribonucleoprotein complex mediates specific DNA cleavage for adaptive immunity in bacteria","volume":"109","author":"G Gasiunas","year":"2012","journal-title":"Proceedings of the National Academy of Sciences of the United States of America"},{"issue":"6096","key":"ref18","doi-asserted-by":"crossref","first-page":"816","DOI":"10.1126\/science.1225829","article-title":"A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity","volume":"337","author":"M Jinek","year":"2012","journal-title":"Science (New York, NY)"},{"issue":"6121","key":"ref19","doi-asserted-by":"crossref","first-page":"819","DOI":"10.1126\/science.1231143","article-title":"Multiplex genome engineering using CRISPR\/Cas systems","volume":"339","author":"L Cong","year":"2013","journal-title":"Science (New York, NY)"},{"issue":"6121","key":"ref20","doi-asserted-by":"crossref","first-page":"823","DOI":"10.1126\/science.1232033","article-title":"RNA-guided human genome engineering via Cas9","volume":"339","author":"P Mali","year":"2013","journal-title":"Science (New York, NY)"},{"key":"ref21","doi-asserted-by":"crossref","first-page":"213","DOI":"10.1016\/j.virol.2015.02.024","article-title":"Bacterial CRISPR\/Cas DNA endonucleases: A revolutionary technology that could dramatically impact viral research and treatment","volume":"479\u2013480","author":"EM Kennedy","year":"2015","journal-title":"Virology"},{"issue":"3","key":"ref22","doi-asserted-by":"crossref","first-page":"759","DOI":"10.1016\/j.cell.2015.09.038","article-title":"Cpf1 is a single RNA-guided endonuclease of a class 2 CRISPR-Cas system","volume":"163","author":"B Zetsche","year":"2015","journal-title":"Cell"},{"issue":"4","key":"ref23","doi-asserted-by":"crossref","first-page":"e1602814","DOI":"10.1126\/sciadv.1602814","article-title":"CRISPR-Cpf1 correction of muscular dystrophy mutations in human cardiomyocytes and mice","volume":"3","author":"Y Zhang","year":"2017","journal-title":"Science advances"},{"key":"ref24","doi-asserted-by":"crossref","first-page":"17","DOI":"10.1016\/j.jconrel.2017.09.012","article-title":"Delivery strategies of the CRISPR-Cas9 gene-editing system for therapeutic applications","volume":"266","author":"C Liu","year":"2017","journal-title":"Journal of controlled release: official journal of the Controlled Release Society"},{"issue":"7","key":"ref25","doi-asserted-by":"crossref","first-page":"e1000097","DOI":"10.1371\/journal.pmed.1000097","article-title":"Preferred reporting items for systematic reviews and meta-analyses: the PRISMA statement","volume":"6","author":"D Moher","year":"2009","journal-title":"PLoS medicine"},{"issue":"10","key":"ref26","doi-asserted-by":"crossref","first-page":"1049","DOI":"10.1038\/ejhg.2012.301","article-title":"Newborn bloodspot screening for Duchenne muscular dystrophy: 21 years experience in Wales (UK)","volume":"21","author":"SJ Moat","year":"2013","journal-title":"European journal of human genetics: EJHG"},{"issue":"8","key":"ref27","doi-asserted-by":"crossref","first-page":"759","DOI":"10.1111\/jpc.12868","article-title":"Duchenne muscular dystrophy","volume":"51","author":"EM Yiu","year":"2015","journal-title":"Journal of paediatrics and child health"},{"issue":"4","key":"ref28","doi-asserted-by":"crossref","first-page":"585","DOI":"10.1016\/j.bbadis.2014.07.023","article-title":"Spectrum of muscular dystrophies associated with sarcolemmal-protein genetic defects","volume":"1852","author":"V Nigro","year":"2015","journal-title":"Biochimica et biophysica acta"},{"issue":"6","key":"ref29","doi-asserted-by":"crossref","first-page":"919","DOI":"10.1016\/0092-8674(87)90579-4","article-title":"Dystrophin: the protein product of the Duchenne muscular dystrophy locus","volume":"51","author":"EP Hoffman","year":"1987","journal-title":"Cell"},{"issue":"6150","key":"ref30","doi-asserted-by":"crossref","first-page":"754","DOI":"10.1038\/330754a0","article-title":"Subcellular fractionation of dystrophin to the triads of skeletal muscle","volume":"330","author":"EP Hoffman","year":"1987","journal-title":"Nature"},{"issue":"2","key":"ref31","doi-asserted-by":"crossref","first-page":"135","DOI":"10.1002\/mus.20586","article-title":"Entries in the Leiden Duchenne muscular dystrophy mutation database: an overview of mutation types and paradoxical cases that confirm the reading-frame rule","volume":"34","author":"A Aartsma-Rus","year":"2006","journal-title":"Muscle & nerve"},{"issue":"2","key":"ref32","doi-asserted-by":"crossref","first-page":"219","DOI":"10.1016\/0092-8674(88)90383-2","article-title":"The complete sequence of dystrophin predicts a rod-shaped cytoskeletal protein","volume":"53","author":"M Koenig","year":"1988","journal-title":"Cell"},{"issue":"3","key":"ref33","doi-asserted-by":"crossref","first-page":"661","DOI":"10.1083\/jcb.135.3.661","article-title":"A new model for the interaction of dystrophin with F-actin","volume":"135","author":"IN Rybakova","year":"1996","journal-title":"The Journal of cell biology"},{"issue":"45","key":"ref34","doi-asserted-by":"crossref","first-page":"28771","DOI":"10.1074\/jbc.272.45.28771","article-title":"Dystrophin-glycoprotein complex is monomeric and stabilizes actin filaments in vitro through a lateral association","volume":"272","author":"IN Rybakova","year":"1997","journal-title":"The Journal of biological chemistry"},{"issue":"5","key":"ref35","doi-asserted-by":"crossref","first-page":"1209","DOI":"10.1083\/jcb.150.5.1209","article-title":"The dystrophin complex forms a mechanically strong link between the sarcolemma and costameric actin","volume":"150","author":"IN Rybakova","year":"2000","journal-title":"The Journal of cell biology"},{"issue":"6","key":"ref36","doi-asserted-by":"crossref","first-page":"1411","DOI":"10.1083\/jcb.118.6.1411","article-title":"Costameres are sites of force transmission to the substratum in adult rat cardiomyocytes","volume":"118","author":"BA Danowski","year":"1992","journal-title":"The Journal of cell biology"},{"issue":"16","key":"ref37","doi-asserted-by":"crossref","first-page":"13591","DOI":"10.1074\/jbc.R200021200","article-title":"Costameres: the Achilles&apos; heel of Herculean muscle","volume":"278","author":"JM Ervasti","year":"2003","journal-title":"The Journal of biological chemistry"},{"key":"ref38","author":"JM E","year":"2000"},{"issue":"418","key":"ref39","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.aan8081","article-title":"Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy","volume":"9","author":"L Amoasii","year":"2017","journal-title":"Science translational medicine"},{"issue":"10","key":"ref40","doi-asserted-by":"crossref","first-page":"1189","DOI":"10.1177\/1087057115586535","article-title":"Drug Discovery of Therapies for Duchenne Muscular Dystrophy","volume":"20","author":"Y Blat","year":"2015","journal-title":"Journal of biomolecular screening"},{"issue":"10","key":"ref41","doi-asserted-by":"crossref","first-page":"871","DOI":"10.1038\/jhg.2017.57","article-title":"Moving towards successful exon-skipping therapy for Duchenne muscular dystrophy","volume":"62","author":"A. Nakamura","year":"2017","journal-title":"Journal of human genetics"},{"issue":"1","key":"ref42","doi-asserted-by":"crossref","first-page":"57","DOI":"10.1089\/nat.2013.0451","article-title":"Skipping multiple exons of dystrophin transcripts using cocktail antisense oligonucleotides","volume":"24","author":"Y Echigoya","year":"2014","journal-title":"Nucleic acid therapeutics"},{"issue":"1","key":"ref43","doi-asserted-by":"crossref","first-page":"9","DOI":"10.1038\/mt.2010.219","article-title":"The status of exon skipping as a therapeutic approach to duchenne muscular dystrophy","volume":"19","author":"QL Lu","year":"2011","journal-title":"Molecular therapy: the journal of the American Society of Gene Therapy"},{"key":"ref44","doi-asserted-by":"crossref","first-page":"6244","DOI":"10.1038\/ncomms7244","article-title":"Multiplex CRISPR\/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy","volume":"6","author":"DG Ousterout","year":"2015","journal-title":"Nature communications"},{"key":"ref45","doi-asserted-by":"crossref","first-page":"11","DOI":"10.1016\/j.omtn.2017.02.004","article-title":"Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR\/Cas9 System","volume":"7","author":"A Lattanzi","year":"2017","journal-title":"Molecular therapy Nucleic acids"},{"issue":"18","key":"ref46","doi-asserted-by":"crossref","DOI":"10.1172\/jci.insight.95918","article-title":"Functional correction of dystrophin actin binding domain mutations by genome editing","volume":"2","author":"V Kyrychenko","year":"2017","journal-title":"JCI insight"},{"issue":"1","key":"ref47","doi-asserted-by":"crossref","first-page":"90","DOI":"10.1016\/j.ajhg.2015.11.012","article-title":"Spell Checking Nature: Versatility of CRISPR\/Cas9 for Developing Treatments for Inherited Disorders","volume":"98","author":"D Wojtal","year":"2016","journal-title":"American journal of human genetics"},{"issue":"3","key":"ref48","doi-asserted-by":"crossref","first-page":"564","DOI":"10.1038\/mt.2015.192","article-title":"CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice","volume":"24","author":"L Xu","year":"2016","journal-title":"Molecular therapy: the journal of the American Society of Gene Therapy"},{"issue":"8","key":"ref49","doi-asserted-by":"crossref","first-page":"923","DOI":"10.1161\/CIRCRESAHA.117.310996","article-title":"In Vivo Genome Editing Restores Dystrophin Expression and Cardiac Function in Dystrophic Mice","volume":"121","author":"M El Refaey","year":"2017","journal-title":"Circulation research"},{"issue":"6271","key":"ref50","doi-asserted-by":"crossref","first-page":"400","DOI":"10.1126\/science.aad5725","article-title":"Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy","volume":"351","author":"C Long","year":"2016","journal-title":"Science (New York, NY)"},{"issue":"6201","key":"ref51","doi-asserted-by":"crossref","first-page":"1184","DOI":"10.1126\/science.1254445","article-title":"Prevention of muscular dystrophy in mice by CRISPR\/Cas9-mediated editing of germline DNA","volume":"345","author":"C Long","year":"2014","journal-title":"Science (New York, NY)"},{"issue":"1","key":"ref52","doi-asserted-by":"crossref","first-page":"108","DOI":"10.1186\/s13059-017-1237-8","article-title":"CRISPR\/Cas9-mediated genome editing induces exon skipping by alternative splicing or exon deletion","volume":"18","author":"H Mou","year":"2017","journal-title":"Genome biology"},{"issue":"6271","key":"ref53","doi-asserted-by":"crossref","first-page":"403","DOI":"10.1126\/science.aad5143","article-title":"In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy","volume":"351","author":"CE Nelson","year":"2016","journal-title":"Science (New York, NY)"},{"issue":"6271","key":"ref54","doi-asserted-by":"crossref","first-page":"407","DOI":"10.1126\/science.aad5177","article-title":"In vivo gene editing in dystrophic mouse muscle and muscle stem cells","volume":"351","author":"M Tabebordbar","year":"2016","journal-title":"Science (New York, NY)"},{"key":"ref55","doi-asserted-by":"crossref","first-page":"31","DOI":"10.1016\/j.omtn.2017.02.007","article-title":"CRISPR\/Cas9-Mediated Genome Editing Corrects Dystrophin Mutation in Skeletal Muscle Stem Cells in a Mouse Model of Muscle Dystrophy","volume":"7","author":"P Zhu","year":"2017","journal-title":"Molecular therapy Nucleic acids"},{"issue":"1","key":"ref56","doi-asserted-by":"crossref","first-page":"143","DOI":"10.1016\/j.stemcr.2014.10.013","article-title":"Precise correction of the dystrophin gene in duchenne muscular dystrophy patient induced pluripotent stem cells by TALEN and CRISPR-Cas9","volume":"4","author":"HL Li","year":"2015","journal-title":"Stem cell reports"},{"issue":"1","key":"ref57","doi-asserted-by":"crossref","first-page":"17113","DOI":"10.1038\/s41598-017-17180-w","article-title":"CRISPR\/Cas9 delivery with one single adenoviral vector devoid of all viral genes","volume":"7","author":"E Ehrke-Schulz","year":"2017","journal-title":"Scientific reports"},{"issue":"3","key":"ref58","doi-asserted-by":"crossref","first-page":"1449","DOI":"10.1093\/nar\/gkv1540","article-title":"Selection-free gene repair after adenoviral vector transduction of designer nucleases: rescue of dystrophin synthesis in DMD muscle cell populations","volume":"44","author":"I Maggio","year":"2016","journal-title":"Nucleic acids research"},{"key":"ref59","doi-asserted-by":"crossref","first-page":"14454","DOI":"10.1038\/ncomms14454","article-title":"Muscle-specific CRISPR\/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy","volume":"8","author":"NE Bengtsson","year":"2017","journal-title":"Nature communications"},{"key":"ref60","doi-asserted-by":"crossref","first-page":"267","DOI":"10.1007\/978-1-4939-7374-3_19","article-title":"From gRNA Identification to the Restoration of Dystrophin Expression: A Dystrophin Gene Correction Strategy for Duchenne Muscular Dystrophy Mutations Using the CRISPR-Induced Deletion Method","volume":"1687","author":"B Duchene","year":"2018","journal-title":"Methods in molecular biology (Clifton, NJ)"},{"key":"ref61","doi-asserted-by":"crossref","first-page":"e283","DOI":"10.1038\/mtna.2015.58","article-title":"Efficient Restoration of the Dystrophin Gene Reading Frame and Protein Structure in DMD Myoblasts Using the CinDel Method","volume":"5","author":"JP Iyombe-Engembe","year":"2016","journal-title":"Molecular therapy Nucleic acids"},{"key":"ref62","doi-asserted-by":"crossref","first-page":"37051","DOI":"10.1038\/srep37051","article-title":"Adenoviral vectors encoding CRISPR\/Cas9 multiplexes rescue dystrophin synthesis in unselected populations of DMD muscle cells","volume":"6","author":"I Maggio","year":"2016","journal-title":"Scientific reports"},{"issue":"4","key":"ref63","doi-asserted-by":"crossref","first-page":"533","DOI":"10.1016\/j.stem.2016.01.021","article-title":"A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells","volume":"18","author":"CS Young","year":"2016","journal-title":"Cell stem cell"},{"issue":"11","key":"ref64","doi-asserted-by":"crossref","first-page":"889","DOI":"10.1038\/s41551-017-0137-2","article-title":"Nanoparticle delivery of Cas9 ribonucleoprotein and donor DNA in vivo induces homology-directed DNA repair","volume":"1","author":"K Lee","year":"2017","journal-title":"Nature Biomedical Engineering"},{"issue":"6201","key":"ref65","doi-asserted-by":"crossref","first-page":"1184","DOI":"10.1126\/science.1254445","article-title":"Prevention of muscular dystrophy in mice by CRISPR\/Cas9-mediated editing of germline DNA","volume":"345","author":"C Long","year":"2014","journal-title":"Science (New York, NY)"},{"issue":"7","key":"ref66","doi-asserted-by":"crossref","first-page":"1495","DOI":"10.1016\/j.cell.2017.10.025","article-title":"In Vivo Target Gene Activation via CRISPR\/Cas9-Mediated Trans-epigenetic Modulation","volume":"171","author":"HK Liao","year":"2017","journal-title":"Cell"},{"key":"ref67","doi-asserted-by":"crossref","first-page":"68","DOI":"10.1016\/j.omtn.2016.11.004","article-title":"Increased Expression of Laminin Subunit Alpha 1 Chain by dCas9-VP160","volume":"6","author":"A Perrin","year":"2017","journal-title":"Molecular therapy Nucleic acids"},{"issue":"1","key":"ref68","doi-asserted-by":"crossref","first-page":"12","DOI":"10.1016\/j.stem.2014.06.011","article-title":"Whole-genome sequencing analysis reveals high specificity of CRISPR\/Cas9 and TALEN-based genome editing in human iPSCs","volume":"15","author":"C Smith","year":"2014","journal-title":"Cell stem cell"},{"issue":"1","key":"ref69","doi-asserted-by":"crossref","first-page":"27","DOI":"10.1016\/j.stem.2014.04.020","article-title":"Low incidence of off-target mutations in individual CRISPR-Cas9 and TALEN targeted human stem cell clones detected by whole-genome sequencing","volume":"15","author":"A Veres","year":"2014","journal-title":"Cell stem cell"},{"issue":"9","key":"ref70","doi-asserted-by":"crossref","DOI":"10.3390\/ijms17091507","article-title":"Biased and Unbiased Methods for the Detection of Off-Target Cleavage by CRISPR\/Cas9: An Overview","volume":"17","author":"F Martin","year":"2016","journal-title":"International journal of molecular sciences"},{"issue":"3","key":"ref71","doi-asserted-by":"crossref","first-page":"827","DOI":"10.1111\/febs.12072","article-title":"Genetic manipulation of myoblasts and a novel primary myosatellite cell culture system: comparing and optimizing approaches","volume":"280","author":"MF Jackson","year":"2013","journal-title":"The FEBS journal"},{"issue":"4","key":"ref72","doi-asserted-by":"crossref","first-page":"950","DOI":"10.1016\/j.vaccine.2009.10.145","article-title":"International epidemiology of human pre-existing adenovirus (Ad) type-5, type-6, type-26 and type-36 neutralizing antibodies: correlates of high Ad5 titers and implications for potential HIV vaccine trials","volume":"28","author":"TC Mast","year":"2010","journal-title":"Vaccine"},{"issue":"11","key":"ref73","doi-asserted-by":"crossref","first-page":"3923","DOI":"10.1182\/blood.V99.11.3923","article-title":"A new adenoviral helper-dependent vector results in long-term therapeutic levels of human coagulation factor IX at low doses in vivo","volume":"99","author":"A Ehrhardt","year":"2002","journal-title":"Blood"},{"issue":"24","key":"ref74","doi-asserted-by":"crossref","first-page":"13565","DOI":"10.1073\/pnas.93.24.13565","article-title":"A helper-dependent adenovirus vector system: removal of helper virus by Cre-mediated excision of the viral packaging signal","volume":"93","author":"RJ Parks","year":"1996","journal-title":"Proceedings of the National Academy of Sciences of the United States of America"},{"issue":"2","key":"ref75","doi-asserted-by":"crossref","first-page":"180","DOI":"10.1038\/ng0298-180","article-title":"Genomic DNA transfer with a high-capacity adenovirus vector results in improved in vivo gene expression and decreased toxicity","volume":"18","author":"G Schiedner","year":"1998","journal-title":"Nature genetics"},{"key":"ref76","doi-asserted-by":"crossref","first-page":"S44","DOI":"10.1016\/S1525-0016(16)32913-6","article-title":"A gene deleted high capacity adenoviral vector for efficient delivery of a multiplex DMD specific CRISPR\/Cas9 machinery","volume":"24","author":"E Ehrke-Schulz","year":"2016","journal-title":"Molecular Therapy"},{"issue":"7","key":"ref77","doi-asserted-by":"crossref","first-page":"547","DOI":"10.1016\/j.braindev.2017.03.024","article-title":"The AAV-mediated and RNA-guided CRISPR\/Cas9 system for gene therapy of DMD and BMD","volume":"39","author":"JZ Wang","year":"2017","journal-title":"Brain & development"},{"issue":"5","key":"ref78","doi-asserted-by":"crossref","first-page":"420","DOI":"10.4161\/cc.2.5.497","article-title":"Cell fusion in skeletal muscle\u2014central role of NFATC2 in regulating muscle cell size","volume":"2","author":"GK Pavlath","year":"2003","journal-title":"Cell cycle (Georgetown, Tex)"},{"issue":"6","key":"ref79","doi-asserted-by":"crossref","first-page":"e1000412","DOI":"10.1371\/journal.pbio.1000412","article-title":"Improving bioscience research reporting: the ARRIVE guidelines for reporting animal research","volume":"8","author":"C Kilkenny","year":"2010","journal-title":"PLoS biology"},{"issue":"10","key":"ref80","doi-asserted-by":"crossref","first-page":"868","DOI":"10.1038\/nmeth.3993","article-title":"A multifunctional AAV-CRISPR-Cas9 and its host response","volume":"13","author":"WL Chew","year":"2016","journal-title":"Nature methods"},{"issue":"5448","key":"ref81","doi-asserted-by":"crossref","first-page":"2244","DOI":"10.1126\/science.286.5448.2244","article-title":"Gene therapy death prompts review of adenovirus vector","volume":"286","author":"E. Marshall","year":"1999","journal-title":"Science (New York, NY)"},{"issue":"4","key":"ref82","doi-asserted-by":"crossref","first-page":"594","DOI":"10.1016\/j.bbadis.2014.05.019","article-title":"Myotonic dystrophies: An update on clinical aspects, genetic, pathology, and molecular pathomechanisms","volume":"1852","author":"G Meola","year":"2015","journal-title":"Biochimica et biophysica acta"},{"key":"ref83","article-title":"Myotonic dystrophy","volume":"2001","author":"P. Harper","year":"2001"},{"issue":"Pt 8","key":"ref84","doi-asserted-by":"crossref","first-page":"1557","DOI":"10.1093\/brain\/121.8.1557","article-title":"Age and causes of death in adult-onset myotonic dystrophy","volume":"121","author":"CE de Die-Smulders","year":"1998","journal-title":"Brain: a journal of neurology"},{"issue":"11","key":"ref85","doi-asserted-by":"crossref","first-page":"974","DOI":"10.1016\/j.nmd.2012.05.017","article-title":"Epidemiological surveillance of myotonic dystrophy type 1: a 25-year population-based study","volume":"22","author":"J Mathieu","year":"2012","journal-title":"Neuromuscular disorders: NMD"},{"issue":"4","key":"ref86","doi-asserted-by":"crossref","first-page":"358","DOI":"10.1136\/jnnp.2008.158261","article-title":"The myotonic dystrophies: diagnosis and management","volume":"81","author":"C Turner","year":"2010","journal-title":"Journal of neurology, neurosurgery, and psychiatry"},{"issue":"5049","key":"ref87","doi-asserted-by":"crossref","first-page":"1253","DOI":"10.1126\/science.1546325","article-title":"Myotonic dystrophy mutation: an unstable CTG repeat in the 3&apos; untranslated region of the gene","volume":"255","author":"M Mahadevan","year":"1992","journal-title":"Science (New York, NY)"},{"issue":"5531","key":"ref88","doi-asserted-by":"crossref","first-page":"864","DOI":"10.1126\/science.1062125","article-title":"Myotonic dystrophy type 2 caused by a CCTG expansion in intron 1 of ZNF9","volume":"293","author":"CL Liquori","year":"2001","journal-title":"Science (New York, NY)"},{"issue":"13","key":"ref89","doi-asserted-by":"crossref","first-page":"2766","DOI":"10.1093\/nar\/29.13.2766","article-title":"In vivo co-localisation of MBNL protein with DMPK expanded-repeat transcripts","volume":"29","author":"M Fardaei","year":"2001","journal-title":"Nucleic acids research"},{"issue":"7","key":"ref90","doi-asserted-by":"crossref","first-page":"805","DOI":"10.1093\/hmg\/11.7.805","article-title":"Three proteins, MBNL, MBLL and MBXL, co-localize in vivo with nuclear foci of expanded-repeat transcripts in DM1 and DM2 cells","volume":"11","author":"M Fardaei","year":"2002","journal-title":"Human molecular genetics"},{"issue":"15","key":"ref91","doi-asserted-by":"crossref","first-page":"3103","DOI":"10.1038\/sj.emboj.7600300","article-title":"Muscleblind proteins regulate alternative splicing","volume":"23","author":"TH Ho","year":"2004","journal-title":"The EMBO journal"},{"issue":"Pt 13","key":"ref92","first-page":"2923","article-title":"Colocalization of muscleblind with RNA foci is separable from mis-regulation of alternative splicing in myotonic dystrophy","volume":"118","author":"TH Ho","year":"2005","journal-title":"Journal of cell science"},{"issue":"3","key":"ref93","doi-asserted-by":"crossref","first-page":"377","DOI":"10.1002\/dvdy.24240","article-title":"Developmental insights into the pathology of and therapeutic strategies for DM1: Back to the basics","volume":"244","author":"A Chau","year":"2015","journal-title":"Developmental dynamics: an official publication of the American Association of Anatomists"},{"issue":"1","key":"ref94","doi-asserted-by":"crossref","first-page":"24","DOI":"10.1016\/j.ymthe.2016.10.014","article-title":"CRISPR\/Cas9-Induced (CTGCAG)n Repeat Instability in the Myotonic Dystrophy Type 1 Locus: Implications for Therapeutic Genome Editing","volume":"25","author":"EL van Agtmaal","year":"2017","journal-title":"Molecular therapy: the journal of the American Society of Gene Therapy"},{"issue":"5","key":"ref95","doi-asserted-by":"crossref","first-page":"899","DOI":"10.1016\/j.cell.2017.07.010","article-title":"Elimination of Toxic Microsatellite Repeat Expansion RNA by RNA-Targeting Cas9","volume":"170","author":"R Batra","year":"2017","journal-title":"Cell"},{"issue":"3","key":"ref96","doi-asserted-by":"crossref","first-page":"479","DOI":"10.1016\/j.molcel.2017.09.033","article-title":"Impeding Transcription of Expanded Microsatellite Repeats by Deactivated Cas9","volume":"68","author":"BS Pinto","year":"2017","journal-title":"Molecular cell"},{"issue":"1","key":"ref97","first-page":"1","article-title":"Genetic basis of limb-girdle muscular dystrophies: the 2014 update","volume":"33","author":"V Nigro","year":"2014","journal-title":"Acta myologica: myopathies and cardiomyopathies: official journal of the Mediterranean Society of Myology"},{"key":"ref98","article-title":"The Limb-Girdle Muscular Dystrophies: Is Treatment on the Horizon?","author":"ML Chu","year":"2018","journal-title":"Neurotherapeutics"},{"issue":"4","key":"ref99","doi-asserted-by":"crossref","first-page":"685","DOI":"10.1038\/mt.2016.40","article-title":"Precise Correction of Disease Mutations in Induced Pluripotent Stem Cells Derived From Patients With Limb Girdle Muscular Dystrophy","volume":"24","author":"S Turan","year":"2016","journal-title":"Molecular therapy: the journal of the American Society of Gene Therapy"},{"issue":"12","key":"ref100","doi-asserted-by":"crossref","first-page":"1056","DOI":"10.1212\/WNL.0000000000000797","article-title":"Population-based incidence and prevalence of facioscapulohumeral dystrophy","volume":"83","author":"JC Deenen","year":"2014","journal-title":"Neurology"},{"issue":"5999","key":"ref101","doi-asserted-by":"crossref","first-page":"1650","DOI":"10.1126\/science.1189044","article-title":"A unifying genetic model for facioscapulohumeral muscular dystrophy","volume":"329","author":"RJ Lemmers","year":"2010","journal-title":"Science (New York, NY)"},{"issue":"3","key":"ref102","doi-asserted-by":"crossref","first-page":"527","DOI":"10.1038\/mt.2015.200","article-title":"CRISPR\/dCas9-mediated Transcriptional Inhibition Ameliorates the Epigenetic Dysregulation at D4Z4 and Represses DUX4-fl in FSH Muscular Dystrophy","volume":"24","author":"CL Himeda","year":"2016","journal-title":"Molecular therapy: the journal of the American Society of Gene Therapy"},{"key":"ref103","doi-asserted-by":"crossref","first-page":"31","DOI":"10.1016\/bs.ctm.2015.05.002","article-title":"Laminin-alpha2 Chain-Deficient Congenital Muscular Dystrophy: Pathophysiology and Development of Treatment","volume":"76","author":"M. Durbeej","year":"2015","journal-title":"Current topics in membranes"},{"issue":"8","key":"ref104","doi-asserted-by":"crossref","first-page":"984","DOI":"10.1038\/nm.4367","article-title":"Correction of a splicing defect in a mouse model of congenital muscular dystrophy type 1A using a homology-directed-repair-independent mechanism","volume":"23","author":"DU Kemaladewi","year":"2017","journal-title":"Nature medicine"},{"issue":"10","key":"ref105","doi-asserted-by":"crossref","first-page":"837","DOI":"10.1016\/S1474-4422(17)30280-6","article-title":"Therapies targeting DNA and RNA in Huntington&apos;s disease","volume":"16","author":"EJ Wild","year":"2017","journal-title":"The Lancet Neurology"},{"issue":"4","key":"ref106","doi-asserted-by":"crossref","first-page":"420","DOI":"10.1097\/WCO.0b013e3283632d90","article-title":"Huntington&apos;s disease and Huntington&apos;s disease-like syndromes: an overview","volume":"26","author":"F Govert","year":"2013","journal-title":"Current opinion in neurology"},{"issue":"6","key":"ref107","doi-asserted-by":"crossref","first-page":"971","DOI":"10.1016\/0092-8674(93)90585-E","article-title":"A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington&apos;s disease chromosomes. The Huntington&apos;s Disease Collaborative Research Group","volume":"72","year":"1993","journal-title":"Cell"},{"key":"ref108","doi-asserted-by":"crossref","first-page":"15005","DOI":"10.1038\/nrdp.2015.5","article-title":"Huntington disease","volume":"1","author":"GP Bates","year":"2015","journal-title":"Nature reviews Disease primers"},{"issue":"20","key":"ref109","first-page":"4566","article-title":"Permanent inactivation of Huntington&apos;s disease mutation by personalized allele-specific CRISPR\/Cas9","volume":"25","author":"JW Shin","year":"2016","journal-title":"Human molecular genetics"},{"issue":"4","key":"ref110","doi-asserted-by":"crossref","DOI":"10.3390\/ijms18040754","article-title":"CRISPR-Cas9 Mediated Gene-Silencing of the Mutant Huntingtin Gene in an In Vitro Model of Huntington&apos;s Disease","volume":"18","author":"N Kolli","year":"2017","journal-title":"International journal of molecular sciences"},{"issue":"3","key":"ref111","doi-asserted-by":"crossref","first-page":"619","DOI":"10.1016\/j.stemcr.2017.01.022","article-title":"Reversal of Phenotypic Abnormalities by CRISPR\/Cas9-Mediated Gene Correction in Huntington Disease Patient-Derived Induced Pluripotent Stem Cells","volume":"8","author":"X Xu","year":"2017","journal-title":"Stem cell reports"},{"issue":"1","key":"ref112","doi-asserted-by":"crossref","first-page":"12","DOI":"10.1016\/j.ymthe.2016.11.010","article-title":"CRISPR\/Cas9 Editing of the Mutant Huntingtin Allele In Vitro and In Vivo","volume":"25","author":"AM Monteys","year":"2017","journal-title":"Molecular therapy: the journal of the American Society of Gene Therapy"},{"issue":"7","key":"ref113","doi-asserted-by":"crossref","first-page":"2719","DOI":"10.1172\/JCI92087","article-title":"CRISPR\/Cas9-mediated gene editing ameliorates neurotoxicity in mouse model of Huntington&apos;s disease","volume":"127","author":"S Yang","year":"2017","journal-title":"The Journal of clinical investigation"},{"key":"ref114","doi-asserted-by":"crossref","first-page":"17065","DOI":"10.1038\/nrdp.2017.65","article-title":"Fragile X syndrome","volume":"3","author":"RJ Hagerman","year":"2017","journal-title":"Nature reviews Disease primers"},{"issue":"2","key":"ref115","doi-asserted-by":"crossref","first-page":"197","DOI":"10.1111\/cge.13075","article-title":"Fragile X syndrome: An overview and update of the FMR1 gene","volume":"93","author":"M Mila","year":"2018","journal-title":"Clinical genetics"},{"issue":"2","key":"ref116","doi-asserted-by":"crossref","first-page":"234","DOI":"10.1016\/j.celrep.2015.08.084","article-title":"Reversion of FMR1 Methylation and Silencing by Editing the Triplet Repeats in Fragile X iPSC-Derived Neurons","volume":"13","author":"CY Park","year":"2015","journal-title":"Cell reports"},{"issue":"10","key":"ref117","doi-asserted-by":"crossref","first-page":"e0165499","DOI":"10.1371\/journal.pone.0165499","article-title":"Reactivation of FMR1 by CRISPR\/Cas9-Mediated Deletion of the Expanded CGG-Repeat of the Fragile X Chromosome","volume":"11","author":"N Xie","year":"2016","journal-title":"PloS one"},{"key":"ref118","doi-asserted-by":"crossref","first-page":"4","DOI":"10.1186\/s40673-017-0062-x","article-title":"Friedreich Ataxia: current status and future prospects","volume":"4","author":"K. Burk","year":"2017","journal-title":"Cerebellum & ataxias"},{"issue":"5","key":"ref119","doi-asserted-by":"crossref","first-page":"1260","DOI":"10.1212\/WNL.47.5.1260","article-title":"Age of onset, sex, and cardiomyopathy as predictors of disability and survival in Friedreich&apos;s disease: a retrospective study on 119 patients","volume":"47","author":"G De Michele","year":"1996","journal-title":"Neurology"},{"issue":"3","key":"ref120","doi-asserted-by":"crossref","first-page":"51","DOI":"10.1097\/NRL.0000000000000054","article-title":"Friedreich Ataxia: From the Eye of a Molecular Biologist","volume":"20","author":"S Muthuswamy","year":"2015","journal-title":"The neurologist"},{"issue":"5","key":"ref121","doi-asserted-by":"crossref","first-page":"265","DOI":"10.1038\/gt.2016.89","article-title":"Deletion of the GAA repeats from the human frataxin gene using the CRISPR-Cas9 system in YG8R-derived cells and mouse models of Friedreich ataxia","volume":"24","author":"DL Ouellet","year":"2017","journal-title":"Gene therapy"},{"issue":"1","key":"ref122","doi-asserted-by":"crossref","first-page":"180","DOI":"10.1016\/j.scr.2015.12.028","article-title":"Generation of an isogenic, gene-corrected control cell line of the spinocerebellar ataxia type 2 patient-derived iPSC line H271","volume":"16","author":"AG Marthaler","year":"2016","journal-title":"Stem cell research"},{"issue":"1","key":"ref123","doi-asserted-by":"crossref","first-page":"162","DOI":"10.1016\/j.scr.2015.12.031","article-title":"Generation of an isogenic, gene-corrected control cell line of the spinocerebellar ataxia type 2 patient-derived iPSC line H196","volume":"16","author":"AG Marthaler","year":"2016","journal-title":"Stem cell research"},{"issue":"1","key":"ref124","doi-asserted-by":"crossref","first-page":"202","DOI":"10.1016\/j.scr.2015.12.048","article-title":"Generation of an isogenic, gene-corrected control cell line of the spinocerebellar ataxia type 2 patient-derived iPSC line H266","volume":"16","author":"AG Marthaler","year":"2016","journal-title":"Stem cell research"},{"issue":"4","key":"ref125","first-page":"625","article-title":"Science and Bioethics of CRISPR-Cas9 Gene Editing: An Analysis Towards Separating Facts and Fiction","volume":"90","author":"AP Cribbs","year":"2017","journal-title":"The Yale journal of biology and medicine"},{"issue":"4","key":"ref126","first-page":"661","article-title":"An Appreciation of The Gene: An Intimate History by Siddhartha Mukherjee and a Call for Expanded Training in the Responsible Conduct of Research","volume":"90","author":"ED Morris","year":"2017","journal-title":"The Yale journal of biology and medicine"},{"issue":"2","key":"ref127","doi-asserted-by":"crossref","first-page":"208","DOI":"10.1111\/bjh.14297","article-title":"CRISPR-Cas9 technology and its application in haematological disorders","volume":"175","author":"H Zhang","year":"2016","journal-title":"British journal of haematology"},{"issue":"1","key":"ref128","doi-asserted-by":"crossref","first-page":"17","DOI":"10.1093\/bmb\/ldx002","article-title":"Ethical issues of CRISPR technology and gene editing through the lens of solidarity","volume":"122","author":"JJ Mulvihill","year":"2017","journal-title":"British medical bulletin"},{"key":"ref129","unstructured":"Maxmen A. (2015) The Genesis Engine. Available: <ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" ext-link-type=\"uri\" xlink:href=\"https:\/\/www.wired.com\/2015\/07\/crispr-dna-editing-2\/\" xlink:type=\"simple\">https:\/\/www.wired.com\/2015\/07\/crispr-dna-editing-2\/<\/ext-link>. Accessed 27 January 2019."},{"key":"ref130","unstructured":"Ball P. (2017) Designer babies: an ethical horror waiting to happen? Available: <ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" ext-link-type=\"uri\" xlink:href=\"https:\/\/www.theguardian.com\/science\/2017\/jan\/08\/designer-babies-ethical-horror-waiting-to-happen\" xlink:type=\"simple\">https:\/\/www.theguardian.com\/science\/2017\/jan\/08\/designer-babies-ethical-horror-waiting-to-happen<\/ext-link>. Accessed 10 October 2018."},{"issue":"12","key":"ref131","doi-asserted-by":"crossref","first-page":"819","DOI":"10.1136\/medethics-2016-103666","article-title":"Everything in moderation, even hype: learning from vaccine controversies to strike a balance with CRISPR","volume":"43","author":"S. Benston","year":"2017","journal-title":"Journal of medical ethics"}],"container-title":["PLOS ONE"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/dx.plos.org\/10.1371\/journal.pone.0212198","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,7,15]],"date-time":"2024-07-15T01:49:29Z","timestamp":1721008169000},"score":1,"resource":{"primary":{"URL":"https:\/\/dx.plos.org\/10.1371\/journal.pone.0212198"}},"subtitle":[],"editor":[{"given":"Alfred S.","family":"Lewin","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"editor"}]}],"short-title":[],"issued":{"date-parts":[[2019,2,22]]},"references-count":131,"journal-issue":{"issue":"2","published-online":{"date-parts":[[2019,2,22]]}},"URL":"https:\/\/doi.org\/10.1371\/journal.pone.0212198","relation":{},"ISSN":["1932-6203"],"issn-type":[{"value":"1932-6203","type":"electronic"}],"subject":[],"published":{"date-parts":[[2019,2,22]]}}}